Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Trikafta

Sionna Therapeutics Inc.

Status and phase: Begins enrollment in 1 month; Phase 2

Conditions: Cystic Fibrosis (CF)

Treatments: Drug: SION-719; Drug: Placebo-to-match SION-719

Study type: Interventional

Funder types: Industry

Identifiers

NCT07108153

SION-719-201

Details and patient eligibility

About

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SION-719 when given to people with CF who are already taking Trikafta.

Enrollment: 16 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of CF with F508del homozygous genotype based on documented CFTR genotype laboratory report.
• Adherent to physician-prescribed Trikafta, as assessed by the Investigator, for at least 3 months prior to the Screening visit, taken at the recommended dose without modifications and on the regimen described in the current prescribing information.
• Stable CF disease without pulmonary exacerbation within 28 days before Baseline or acute non-CF-related illness within 14 days before Baseline.

Exclusion criteria

• Participant has clinically significant current or recurrent illness, other than CF
• Participant has a history of malignancy, except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of recurrence for at least 1 year.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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16 participants in 2 patient groups

Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719

Experimental group

Description:

Participants receive SION-719 for one treatment period, and placebo to match SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study.

Treatment:

Drug: Placebo-to-match SION-719

Drug: SION-719

Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719

Experimental group

Description:

Participants receive placebo to match SION-719 for one treatment period and SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study.

Treatment:

Drug: Placebo-to-match SION-719

Drug: SION-719